Breakthrough Tx Status Granted to Treatment for Rare Obesity Disorder

Rhythm announced that the Food and Drug Administration (FDA) has granted setmelanotide Breakthrough Therapy designation for the treatment of pro-opiomelanocortin (POMC) deficiency obesity, and Orphan Drug designation for the treatment of Prader-Willi Syndrome (PWS).

POMC deficiency obesity is a rare, genetic disorder that results in extreme and unrelenting appetite and obesity due to impaired melanocortin-4 (MC4) pathway function. PWS is a rare, life‑threatening disease characterized by severe hyperphagia, an overriding physiological drive to eat, leading to severe obesity and other complications. Currently, there is no approved treatment for PWS-associated obesity and hyperphagia.